JP2022507534A5 - - Google Patents
Info
- Publication number
- JP2022507534A5 JP2022507534A5 JP2021526562A JP2021526562A JP2022507534A5 JP 2022507534 A5 JP2022507534 A5 JP 2022507534A5 JP 2021526562 A JP2021526562 A JP 2021526562A JP 2021526562 A JP2021526562 A JP 2021526562A JP 2022507534 A5 JP2022507534 A5 JP 2022507534A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- molecule according
- factor
- expression vector
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862768744P | 2018-11-16 | 2018-11-16 | |
| US62/768,744 | 2018-11-16 | ||
| PCT/US2019/061808 WO2020102723A1 (en) | 2018-11-16 | 2019-11-15 | Compositions and methods for treating wilson's disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022507534A JP2022507534A (ja) | 2022-01-18 |
| JP2022507534A5 true JP2022507534A5 (https=) | 2023-06-27 |
| JP7532360B2 JP7532360B2 (ja) | 2024-08-13 |
Family
ID=70731690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021526562A Active JP7532360B2 (ja) | 2018-11-16 | 2019-11-15 | ウィルソン病を処置するための組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210369870A1 (https=) |
| EP (2) | EP3880809A4 (https=) |
| JP (1) | JP7532360B2 (https=) |
| CN (1) | CN113302290A (https=) |
| CA (1) | CA3118936A1 (https=) |
| TW (1) | TW202039854A (https=) |
| WO (1) | WO2020102723A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112019013245A2 (pt) | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| DK3906066T5 (da) | 2019-01-04 | 2024-08-05 | Ultragenyx Pharmaceutical Inc | Geneterapikonstruktioner til behandling af wilsons sygdom |
| US20240307553A1 (en) * | 2021-04-01 | 2024-09-19 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| CN115216481A (zh) * | 2021-04-21 | 2022-10-21 | 四川至善唯新生物科技有限公司 | 一种表达量提高的截短型atp7b基因及其应用 |
| CN113699211A (zh) * | 2021-07-20 | 2021-11-26 | 深圳市儿童医院 | 一种用于儿童威尔森氏症快速筛查的参数组合和试剂盒 |
| WO2023038909A1 (en) * | 2021-09-07 | 2023-03-16 | Alexion Pharmaceuticals, Inc. | Methods of treating copper metabolism-associated diseases or disorders |
| TW202323528A (zh) * | 2021-10-18 | 2023-06-16 | 美商邏輯生物療法公司 | 用於治療威爾森氏病之基因療法 |
| JP2025533994A (ja) * | 2022-10-11 | 2025-10-09 | メイラグティーエックス ユーケー アイアイ リミティド | Ａｔｐ７ｂ遺伝子療法 |
| EP4677077A1 (en) * | 2023-03-06 | 2026-01-14 | Lingyi Biotech Co., Ltd. | A truncated atp7b and the use thereof |
| WO2024193638A1 (en) * | 2023-03-22 | 2024-09-26 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of wilson disease |
| WO2025002226A1 (zh) * | 2023-06-28 | 2025-01-02 | 迈威(上海)生物科技股份有限公司 | 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用 |
| CN120882865A (zh) * | 2023-07-28 | 2025-10-31 | 凌意(杭州)生物科技有限公司 | 用于治疗威尔逊病的多核苷酸 |
| CN117757797B (zh) * | 2023-09-25 | 2024-08-16 | 北京圭特细胞科技有限公司 | 一种编码人III型胶原蛋白α链的核酸及其应用 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
| CN121265551A (zh) * | 2024-07-02 | 2026-01-06 | 苏州星核迪赛生物技术有限公司 | 一种靶向肝实质细胞的核酸递送载体及其应用 |
| CN121319202B (zh) * | 2025-12-15 | 2026-04-07 | 凌意(杭州)生物科技有限公司 | Atp7b抗体及其应用 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ES2284545T3 (es) * | 1999-11-16 | 2007-11-16 | Genzyme Corporation | Vectores y transgenes con elementos reguladores para la administracion de genes del higado. |
| EP1103610A1 (en) | 1999-11-26 | 2001-05-30 | Introgene B.V. | Production of vaccines from immortalised mammalian cell lines |
| FR2808803B1 (fr) | 2000-05-11 | 2004-12-10 | Agronomique Inst Nat Rech | Cellules es modifiees et gene specifique de cellules es |
| FR2836924B1 (fr) | 2002-03-08 | 2005-01-14 | Vivalis | Lignees de cellules aviaires utiles pour la production de substances d'interet |
| US7261544B2 (en) | 2003-05-21 | 2007-08-28 | Genzyme Corporation | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1528101A1 (en) | 2003-11-03 | 2005-05-04 | ProBioGen AG | Immortalized avian cell lines for virus production |
| FR2884255B1 (fr) | 2005-04-11 | 2010-11-05 | Vivalis | Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe |
| US8048999B2 (en) | 2005-12-13 | 2011-11-01 | Kyoto University | Nuclear reprogramming factor |
| JP5813321B2 (ja) | 2007-03-23 | 2015-11-17 | ウィスコンシン アラムニ リサーチ ファンデーション | 体細胞の再プログラム化 |
| EP1985305A1 (en) | 2007-04-24 | 2008-10-29 | Vivalis | Duck embryonic derived stem cell lines for the production of viral vaccines |
| EP1995309A1 (en) | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
| KR20180000341A (ko) | 2009-06-16 | 2018-01-02 | 젠자임 코포레이션 | 재조합 ａａｖ 벡터에 대한 개선된 정제 방법 |
| WO2012097826A2 (en) | 2011-01-21 | 2012-07-26 | Aarhus Universitet | Crystal structure of a type ib p-type atpase |
| EP3105334B1 (en) * | 2014-02-11 | 2019-07-03 | Friedrich Miescher Institute for Biomedical Research | Müller cell-specific promoter |
| US11118238B2 (en) * | 2014-12-17 | 2021-09-14 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions |
| US11147887B2 (en) * | 2014-12-17 | 2021-10-19 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease |
| EP3240558A1 (en) * | 2014-12-30 | 2017-11-08 | CureVac AG | Artificial nucleic acid molecules |
| US11007280B2 (en) * | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| GB201522416D0 (en) | 2015-12-18 | 2016-02-03 | Ucl Business Plc | Wilson's disease gene therapy |
| BR112019013245A2 (pt) * | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
-
2019
- 2019-11-15 EP EP19884231.2A patent/EP3880809A4/en not_active Withdrawn
- 2019-11-15 EP EP23185569.3A patent/EP4257155A3/en active Pending
- 2019-11-15 CN CN201980089235.3A patent/CN113302290A/zh active Pending
- 2019-11-15 WO PCT/US2019/061808 patent/WO2020102723A1/en not_active Ceased
- 2019-11-15 US US17/294,325 patent/US20210369870A1/en not_active Abandoned
- 2019-11-15 JP JP2021526562A patent/JP7532360B2/ja active Active
- 2019-11-15 TW TW108141687A patent/TW202039854A/zh unknown
- 2019-11-15 CA CA3118936A patent/CA3118936A1/en active Pending
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